RARE Daily

FDA approves Nobelpharma’s Hyftor Topical Treatment for Facial Angiofibromas in People with TSC

April 4, 2022

The U.S. Food and Drug Administration approved Nobelpharma’s Hyftor as the first topical treatment indicated for facial angiofibroma associated with tuberous sclerosis complex in adults and children six years of age or older.

Photo: Yoshiki Kida, president and CEO of Nobelpharma America

Tuberous sclerosis complex (TSC) is a rare genetic disorder that causes non-cancerous tumors to form in various organs, primarily the brain, eyes, heart, kidneys, skin, and lungs, and continues over the lifetime of those who have it. Facial angiofibroma is seen in approximately 75 percent to 80 percent of TSC patients. Facial angiofibromas associated with TSC refer to facial skin lesions caused by genetic mutations resulting in unregulated cell growth. Without treatment, these may cause significant disfiguration, bleeding, pruritus, and erythema. In the U.S., approximately 50,000 people have TSC, and an estimated 40,000 have TSC-related facial angiofibroma, with most diagnosed as children. No difference in prevalence has been observed by gender, race, or ethnicity.

“The U.S. approval of Hyftor is a significant milestone for Nobelpharma. Not only is this our first FDA approval, but it is also the first ever FDA-approved topical treatment for facial angiofibroma associated with tuberous sclerosis complex,” said Yoshiki Kida, president and CEO of Nobelpharma America. “This landmark approval will bring a much-needed therapy to people with tuberous sclerosis complex.”

Hyftor (sirolimus topical gel) 0.2% is the first and only clear topical medication approved by the FDA to treat facial angiofibroma associated with TSC in adults and children 6 years of age and older. In clinical studies, Hyftor has been shown to improve the size and redness of facial angiofibromas at 12 weeks. Hyftor is contraindicated in patients with a history of hypersensitivity to sirolimus or any other component of the gel.

Author: Rare Daily Staff

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